Capricor Reports 4-Year Data For Deramiocel, Shows Sustained Benefit In Duchenne Patients

Capricor Therapeutics (NASDAQ:CAPR) on Friday released four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD).

After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline.

Further, a subgroup analysis of patients with baseline Left Ventricular Ejection Fraction (LVEF) >45% showed an even greater clinical benefit, supporting early intervention with Deramiocel to potentially preserve cardiac function.

Also Read: Capricor Therapeutics Cell Therapy Goes Under Priority FDA Review For Duchenne Associated-Heart Condition

Additionally, treatment continued to slow skeletal muscle disease progression, as measured by performance of the upper limb (PUL v2.0), with patients experiencing a smaller average decline in the fourth year (0.6 points) compared to the first year (1.8 points).

Together, these findings suggest that extended treatment with Deramiocel may help attenuate the progression of DMD over time. Deramiocel continues to maintain a favorable safety profile throughout the study.

“These four-year data reinforce the strength and durability of Deramiocel’s clinical benefit and favorable safety profile across both cardiac and skeletal muscle function,” said Linda Marbán, CEO of Capricor Therapeutics. “With our BLA under priority review and several key regulatory steps now completed, we are executing with focus and urgency as we move toward potential approval.

CAPR Price Action: CAPR stock is down 31.75% at $8.16 at publication on Friday.

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